Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by mutations in the DMD gene that prevent the expression of a functional dystrophin protein. Exon duplications represent 6%–11% of mutations, and duplications of exon 2 (Dup2) are the most common (∼11%) of duplication mutatio...

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Bibliographic Details
Main Authors: Liubov V. Gushchina, Adrienne J. Bradley, Tatyana A. Vetter, Jacob W. Lay, Natalie L. Rohan, Emma C. Frair, Nicolas Wein, Kevin M. Flanigan
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Duchenne muscular dystrophy
Becker muscular dystrophy
dystrophin
exon skipping
U7snRNA
scAAV9.U7.ACCA
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001833

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http://www.sciencedirect.com/science/article/pii/S2329050123001833

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